Key Points of the PDUFA V Reauthorization Technical Letter

The FDA and industry have reached an agreement on the reauthorization of the Prescription Drug User Fee Act (PDUFA V) -- the first major milestone in the reauthorization process. After reading the technical letter posted on the FDA website, we thought this would be a good opportunity to break down and explain some of the important points of the agreement.

New Review Model

The technical agreement includes an enhanced model for reviewing new drug applications. The new review model will extend submission timelines by two months and allow additional meetings between industry and the FDA periodically throughout the review process. These meetings will hopefully foster better and more open communication on drug applications and provide an opportunity to raise potential issues earlier in the review process.

Enhancements to the Drug Review Process

Industry agreed to fund several improvements to the drug review process by increasing user fees. These new resources will pay for new staff, training, public meetings and guidance to industry, will improve FDA's regulatory science, and better equip the FDA to more adequately provide a timely review for new medicines. These provisions are detailed below.

Enhancing Benefit-Risk Assessment in the Drug Review Process

The agreement contains provisions that will enhance benefit-risk assessment in regulatory decision-making and includes a concentration on patient-focused drug development. Patients remain strong advocates for new drug development and their voices can be helpful during the FDA review process. This provision is part of a solution that should produce more timely, balanced and higher quality decisions by the FDA.

Advancing the Development of Drugs for Rare Diseases

Advancing development of medicines for rare or orphan diseases is important to the nearly 25 million patients who have a disease that is considered "rare." Many rare diseases have unmet medical needs with no approved medical therapies. The PDUFA V technical agreement contains provisions that will improve the outreach efforts to the rare disease patient community, and provide specialized training in rare disease drug development for sponsors and FDA staff to help bring new cures and treatments to the patients that need them.

Evaluating Post-Market Drug Safety

Currently, the medical monitoring system includes voluntary reporting of medical adverse events by patients, health care providers, and other stakeholders to provide a collection of information regarding the safety of a drug after it is on the market. The FDA would like to enhance the Sentinel Initiative which has the potential to give both the FDA and industry additional capability to provide individual patients the best information possible about new medicines to ensure that they can make an appropriate and informed decision with their health care provider. We wrote more about the Sentinel Initiative earlier in the summer when it was first raised.

Developments in Pharmacogenomics and Biomarkers

A biomarker is a substance used in clinical trials to show changes in biological processes, or responses to treatments. The FDA provides regulatory advice on the use of biomarkers to assess safety and efficacy in clinical studies. The PDUFA V enhancement would bolster the FDA's capacity to adequately address submissions that propose to utilize biomarkers or pharmacogenomics.

Developing New Methods for Meta-Analysis

A meta-analysis typically attempts to combine the data or findings from multiple completed studies to explore drug benefits and risks and, in some cases, uncover what might be a potential safety signal. The new agreement includes additional funding to help the FDA develop new tools to validate or reject the findings from meta-analyses. This will help eliminate uncertainty for patients and health care providers.

Improving the Use of Patient-Reported Outcomes

A Patient-reported outcome (PRO) is a self-report about treatment results collected directly from a patient via a questionnaire during a clinical trial. Effectively measuring PROs is critical in understanding the drug benefits and harm from the patients' perspective. PDUFA V enhancements would improve FDA's clinical and statistical ability to address submissions involving PROs -- including providing consultation during the early stages of drug development which will help ensure that medicine can efficiently reach patients in need.

Promoting Innovation Through Enhanced Communication Between FDA and Industry During Drug Development

FDA recognizes that timely and interactive communication with sponsors can help foster efficient and effective drug development. The agreement includes several proposals to fund an agency communication and training staff focused on improving communication between FDA and sponsors during development.

Standardizing Risk Evaluation and Management Strategies (REMS)

FDAAA gave FDA authority to require REMS which have created burdens on the health care system. A PDUFA V enhancement would aim to standardize REMS with the goal of reducing burden on practitioners, patients, and others in the health care setting.

Electronic Submissions and Standardization of Electronic Application Data

The FDA currently receives sponsor submissions of original applications and supplements in a variety of different formats. This can present obstacles to conducting a timely, efficient, and rigorous review within current PDUFA goal time frames. The technical agreement includes a phased-in requirement for standardized, fully electronic application submissions. This will ensure the FDA can devote more time to drug approvals and less to digitizing paper submissions or standardizing data sets.

We believe that this technical letter is a great first step to a clean PDUFA. A clean PDUFA is one that helps return to a regulatory system that is better funded and holds the FDA accountable for speeding safe, new treatments to patients. As the technical letter moves to Congress for reauthorization, the Campaign will continue to remind Congress of why PDUFA was passed in the first place and how, if functioning as intended, it brings immense benefits to patients and America alike.